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Gene Therapy Trial Report

Summary

A Study to Assess the Safety, Tolerability, and Efficacy of ST-400 for Treatment of Transfusion-Dependent Beta-thalassemia (TDT)


NCTID NCT03432364 (View at clinicaltrials.gov)
Description
Development Status Inactive
Indication Beta-Thalassemia Major
Disease Ontology Term DOID:0080771
Compound Name ST-400
Sponsor Sangamo Therapeutics
Funder Type Industry
Recruitment Status
Completed
Enrollment Count 5 (ACTUAL)
Results Posted View Results

Therapy Information


Target Gene/Variant BCL11A
Therapy Type Gene editing
Therapy Route Ex-vivo
Mechanism of Action Overexpression of protective allele/gene
Route of Administration Intravenous
Drug Product Type Autologous cells
Target Tissue/Cell CD34+ cells
Delivery System Electroporation
Editor Type ZFN mRNA
Dose 1 Minimum dose: 4.5E6 cells/kg
Dose 2 Max dose: 11.4E6 CD34+ cells/kg
Dose 3 Average dose: 7.3E6 CD34+ cells/kg

Study Record Dates


Current Stage Phase1, Phase2
Submit Date 2018-02-01
Completion Date 2022-11-17
Last Update 2023-12-14

Participation Criteria


Eligible Age 18 Years - 40 Years
Standard Ages Adult
Sexes Eligible for Study ALL
Eligibility Criteria
Inclusion Criteria: 1. Informed Consent 2. Clinical diagnosis of TDT with ≥ 8 documented RBC transfusion events per year on an annualized basis in the 2-years prior to screening 3. Confirmed beta-thalassemia diagnosis by molecular genetic testing 4. Clinically stable and eligible to receive conditioning chemotherapy 5. Able and willing to use an effective method of contraception from the signing of the informed consent and for one year following ST-400 infusion. Exclusion Criteria: 1. Previous history of autologous or allogeneic blood stem cell transplantation or solid organ transplantation 2. Pregnant or breastfeeding female 3. Medical contraindication to mobilization, apheresis, or conditioning 4. Significant liver, lung, heart, or kidney dysfunction 5. Diagnosis of HIV or evidence of active HBV or HCV 6. History of significant bleeding disorder or uncontrolled seizures 7. History of active malignancy in past 5 years (non-melanoma skin cancer or cervical cancer in situ permitted) any history of hematologic malignancy, or family history of cancer predisposition syndrome without negative testing result in the study candidate. 8. Currently participating in another clinical trial using an investigational study medication, or recent participation in such a trial 9. Previous treatment with gene therapy
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 6
Locations United States

Regulatory Information


Has US IND True
FDA Designations Orphan Drug Designation
Recent Updates Development discontinued by Sponsor November 2021

Resources/Links